This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Clinical Trial Characterizing the Bioavailability of 1-Octanol in Adults With Ethanol-responsive Essential Tremor (NCT00102596), a na phase 2 study. The primary outcome is Normalized Mean Tremor Amplitude for Both Formulations of 64 mg/kg 1-Octanol in Part B.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00102596 |
| Phase | Phase 2 |
| Condition | Essential Tremor |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 21 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Institute of Neurological Disorders and Stroke (NINDS) |
| Results posted | Yes |
2. Clinical Question
Population
Adults with ethanol-responsive essential tremor. The registry lists an enrollment of 21, but the number of participants in the primary-outcome population is not reported in the registry record.
Intervention
1-octanol, with the primary outcome describing both formulations at 64 mg/kg in Part B. The study also aimed to assess drug and metabolite bioavailability.
Comparator
The primary outcome concerns both formulations in the same study population. The record does not describe a separate placebo or untreated control group, and it does not present a randomized comparison.
Primary question
How does mean normalized tremor amplitude, measured by spirography after dosing, vary over time for the two formulations? A normalized score of 1 is baseline severity; lower scores indicate tremor reduction. The numerical outcome results are not reported in the registry record.
The clinical question is about tremor measurements and formulation-related bioavailability in adults whose essential tremor is described as ethanol-responsive. The registry states that tremor amplitude was measured in the right hand and normalized to each participant’s baseline severity. This makes the outcome a relative measure: it expresses each recorded score in relation to that participant’s baseline rather than in the original measurement units.
The primary outcome lists measurements at 0, 15, 30, 60, 90, 120, 150, 180, 240 and 360 minutes post-dose. The record identifies a single-group model and no masking. It does not describe a randomized between-group treatment comparison. The results available in the registry record do not give the mean normalized scores or their standard errors, so the pattern and precision of tremor measurements cannot be assessed from the record.
3. Trial Design
The registry identifies this as a Phase 2, completed, unblinded study with a single-group model. Enrollment is listed as 21. The brief study description calls it a two-phase inpatient study, but the available description is incomplete and does not detail the procedures for each phase. Allocation is recorded as not applicable, and masking is listed as none.
The intervention is 1-octanol. The primary outcome concerns both formulations of 64 mg/kg in Part B, and the defined primary-outcome population consists of participants who received both formulations in Part B. The number of participants meeting that definition is not reported in the registry record. Although measurements following the formulations may permit within-participant comparisons, the record does not describe a randomized control arm or a like-for-like comparison with an independent group.
The study objective included evaluating formulation efficacy using accelerometry and spirography and evaluating drug and metabolite bioavailability in plasma and urine using high performance liquid chromatography. The listed secondary outcomes include plasma levels of octanoic acid after a 64 mg/kg 1-octanol dose, heart rate after dosing, and PR and QTc intervals after dosing.
These design details establish what was intended to be studied, but they do not establish what the observed results were. Numerical primary-outcome estimates, the number analyzed for that outcome, and a detailed account of the phase-specific procedures are not reported in the registry record. The protocol, detailed statistical plan, and sample-size justification are also not reported in the registry record. The planning illustration is not evidence of the trial’s original design or of the number of participants needed for this study.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Part A
| Stage | Part A: Dose Escalation | Part B Then C: Fixed Dose |
|---|---|---|
| STARTED | 11 | 0 |
| Participants Assigned Treatment | 5 | 0 |
| COMPLETED | 4 | 0 |
| NOT COMPLETED | 7 | 0 |
Reasons for not completing this period:
| Reason | Part A: Dose Escalation | Part B Then C: Fixed Dose |
|---|---|---|
| Screen failures | 6 | 0 |
| Adverse Event | 1 | 0 |
Part B
| Stage | Part A: Dose Escalation | Part B Then C: Fixed Dose |
|---|---|---|
| STARTED | 0 | 10 |
| COMPLETED | 0 | 10 |
| NOT COMPLETED | 0 | 0 |
Part C
| Stage | Part A: Dose Escalation | Part B Then C: Fixed Dose |
|---|---|---|
| STARTED | 0 | 2 |
| COMPLETED | 0 | 2 |
| NOT COMPLETED | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: All participants who received both formulations of 64 mg/kg 1-octanol in Part B
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | All Participants |
|---|---|---|
| Participants analysed | 15 | |
| Age Continuous | years | 68.1 (SD 8.1) |
| Sex: Female, Male: Female | Participants | 6 |
| Sex: Female, Male: Male | Participants | 9 |
| Region of Enrollment: United States | participants | 15 |
| Height | centimeters | 169.6 (SD 12.0) |
| Weight | kg | 82.9 (SD 20.1) |
| Body Mass Index | kg/m^2 | 28.6 (SD 4.7) |
| Age of onset | years | 39.5 (SD 18.6) |
| Disease Duration | years | 28.7 (SD 17.2) |
| History of alcohol-responsive tremors | participants | 15 |
| Number of alcohol servings required for tremor response | servings | 2.1 (SD 0.3) |
| Family history of essential tremor | participants | 13 |
| Fahn Tolosa Marin Tremor Rating Scale | scores on a scale | 41.6 (SD 17.6) |
6. Primary Endpoint
The registry lists the primary outcome as Normalized Mean Tremor Amplitude for Both Formulations of 64 mg/kg 1-Octanol in Part B, measured over 0, 15, 30, 60, 90, 120, 150, 180, 240 and 360 minutes post-dose.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Error |
| Unit | normalized score on a scale |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is the mean normalized tremor amplitude for both formulations of 64 mg/kg 1-octanol in Part B. Spirography measured tremor amplitude in the right hand at 0, 15, 30, 60, 90, 120, 150, 180, 240 and 360 minutes post-dose. Each participant’s measurements were divided by that participant’s baseline tremor severity score. The resulting normalized score uses 1 to represent baseline; lower values indicate tremor reduction.
Normalization can make measurements easier to interpret relative to a participant’s own starting severity, rather than as raw amplitude alone. It does not by itself show whether a change is clinically important, whether it is attributable to the study drug, or whether the response differs between formulations. Those questions require the observed estimates, their uncertainty, and an analysis suited to the study design.
The endpoint is reported as a mean with standard error. A mean describes the average normalized score in the defined analysis population. The standard error describes the precision of that estimated mean, not the spread of individual participants’ measurements. The actual means and standard errors are not reported in the registry record, so the degree of tremor change and its precision cannot be described from the posted results.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record defines a tremor outcome that is interpretable in relation to baseline: a normalized score of 1 corresponds to baseline tremor severity, and lower scores indicate tremor reduction. However, the registry record does not report the mean scores, standard errors, or other numerical primary-outcome results at the listed measurement times. It therefore does not establish the magnitude, timing, or precision of any observed change in tremor amplitude.
The primary population is described as all participants who received both formulations in Part B. This is a specific analysis population, not necessarily the same as everyone enrolled. The number of participants in that population and the number contributing data at each time point are not reported in the registry record. Without those details, readers cannot assess how much information supports the endpoint or whether missing measurements affected the analysis.
The single-group, unblinded design also shapes what can be concluded. Measurements across formulations or over time can describe what was observed among participants studied, but without a randomized control comparison they do not isolate the effect of 1-octanol from other explanations. The record does not report a statistical contrast between formulations, nor does it provide a test result or confidence interval for the primary endpoint.
General statistical context: a confidence interval, when reported, conveys a range of values compatible with the data under the analysis assumptions and helps show estimate precision. A standard error is one component used to describe that precision, but a standard error without the underlying estimate does not show the direction or size of a result. Neither measure is reported numerically for the primary endpoint in the registry record.
Consequently, no claim about statistical significance, formulation differences, or the extent of tremor reduction can be made from the posted results. The record’s stated objective and endpoint describe what investigators set out to measure; they are not substitutes for outcome estimates. The sponsor’s own analysis results are not reported in the registry record, and no recalculated estimates are available to interpret.
9. Statistical Methods Explained
What does the mean represent?
The primary outcome is a mean of a continuous measure. Each normalized score represents tremor amplitude relative to the participant’s baseline severity, with 1 denoting baseline and lower scores denoting tremor reduction. A mean combines the participant-level values into an average for the specified analysis population. It can provide a concise description of the group’s measured response, but it does not show how similar or different individual responses were.
The listed time points describe when measurements were scheduled after dosing. Measurements from the same participant over time are related observations, rather than independent observations. General statistical context: analyses of repeated measurements should account for that dependence when estimating changes over time or comparing patterns. The registry record does not report the statistical model used for the primary outcome, so it is not possible to assess how such dependence was handled.
How should the standard error be read?
The primary endpoint specifies standard error as its dispersion measure. General statistical context: standard error describes the precision of an estimated mean across repeated samples under a statistical model. It is not the same as the standard deviation, which describes variation among individual observations. A small standard error can indicate a more precisely estimated mean, but interpretation also requires the mean itself and an understanding of the analysis population and assumptions.
The numerical means and standard errors are not reported in the registry record. As a result, readers cannot judge the estimate’s precision or compare the endpoint across the listed times. There is also no reported confidence interval or inferential test for the primary outcome.
What does normalization contribute?
Dividing each participant’s tremor amplitude by that participant’s baseline severity expresses follow-up measurements as a proportion of baseline. This places measurements on a relative scale and gives the baseline a defined reference value of 1. A lower normalized score indicates tremor reduction according to the endpoint description. Normalization does not remove all differences among participants, nor does it establish that an observed change was caused by treatment. Interpretation still depends on the study design, the observed values, and the analysis method.
What can the planning illustration tell readers?
The planning illustration is described as a calculation based on the observed effect, not as the trial’s original design. Its assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the target effect is described as a stated fraction of the observed effect. For non-time-to-event outcomes, the illustration expresses sample sizes as participants per arm for 80% power or other stated power levels. These are planning illustrations, not observed power and not a reconstruction of the trial’s design.
General statistical context: power calculations estimate the sample size needed to detect a specified effect under stated assumptions. They depend on the effect size, variability, significance threshold, allocation, and analysis model. Calculations that use an effect estimated from a completed study are illustrative because that effect may itself be uncertain. The observed primary effect and numerical planning outputs are not reported in the registry record, so the illustration cannot be independently interpreted as evidence about the study’s ability to detect an effect.
What remains unknown about the analysis?
The registry record does not report the primary statistical model, the planned handling of repeated measurements, a confidence interval method, or the test used for any comparison. It also does not report whether analyses adjusted for baseline or other covariates. These details matter because modeling choices can affect estimates and uncertainty, especially when observations are repeated within participants. Without the analysis plan and numerical results, the assumptions behind any claimed effect cannot be evaluated from the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists the normalized tremor-amplitude outcome as primary and lists secondary outcomes concerning plasma octanoic acid levels, heart rate, and PR and QTc intervals. The primary outcome itself includes measurements at multiple post-dose times and concerns both formulations. The registry record does not report how comparisons across times, formulations, or outcomes were handled, or whether any multiplicity adjustment was planned. Without that information, the chance of false-positive findings across multiple statistical tests cannot be assessed.
The registry defines the primary-outcome population as all participants who received both formulations in Part B. It does not report how many participants met that definition, how many contributed an observation at each time, or whether the analysis used all available measurements. The missing-data method, reasons for missing measurements, and any sensitivity analyses are not reported in the registry record.
These omissions limit interpretation because a defined analysis population and missing-data strategy clarify which observations support an estimate and how incomplete follow-up is addressed. The registry record also does not state whether participants were analyzed according to an intention-to-treat principle or another named approach. No inference about those procedures can be made from the stated population definition alone.
The number of statistical analyses listed in the registry record is zero. That does not establish that investigators performed no analyses; it means the registry record does not provide analysis entries to evaluate. Numerical outcome estimates and a detailed statistical plan are not reported in the registry record.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| 1-octanol Dose | 2 | 15 | 13.3% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| 1-octanol Dose | 14 of 15 |
Most frequent serious adverse event terms
| Event term | Organ system | 1-octanol Dose |
|---|---|---|
| Fever | Infections and infestations | 1 of 15 |
| Fall | Injury, poisoning and procedural complications | 1 of 15 |
Most frequent non-serious adverse event terms
| Event term | Organ system | 1-octanol Dose |
|---|---|---|
| Taste Change | Gastrointestinal disorders | 8 of 15 |
| Headache | General disorders | 5 of 15 |
| Heartburn | Gastrointestinal disorders | 5 of 15 |
| Gas/Bloating | Gastrointestinal disorders | 5 of 15 |
| Nausea | Gastrointestinal disorders | 4 of 15 |
| Dry Mouth | Gastrointestinal disorders | 4 of 15 |
| Constipation | Gastrointestinal disorders | 3 of 15 |
| Sedation | General disorders | 2 of 15 |
| Dizziness | General disorders | 2 of 15 |
| Cough | Respiratory, thoracic and mediastinal disorders | 2 of 15 |
Serious events with the highest arm-level rate: fever (6.7%), fall (6.7%).
The registry lists a 1-octanol Dose group with 15 participants at risk for serious adverse events and 2 affected. It lists 15 at risk for other adverse events and 14 affected. Fever and fall are each listed among the serious adverse-event terms at 6.7%. These figures describe the registry’s safety entries; they do not by themselves establish that an event was caused by 1-octanol.
The at-risk and affected figures should be read as separate safety summaries, not as measures of the primary tremor outcome. The record does not provide event-level timing, severity details beyond the serious classification, or an account of causal assessment. The death figure is not reported in the registry record.
Safety counts are descriptive. They do not establish comparative safety because the record does not describe a randomized control group. They also do not characterize the risk for people outside the study population. The study’s unblinded, single-group design and the available registry entries should be kept in mind when interpreting these reports. No conclusion about the overall safety profile can be drawn from these figures alone.
12. What the Result Does, and Does Not, Mean
The record is useful for identifying the study population, intervention, design, and intended outcome. It describes adults with ethanol-responsive essential tremor, a Phase 2 unblinded single-group study, and a primary measure based on tremor amplitude normalized to baseline. It also records safety counts and secondary measures related to bioavailability and cardiovascular monitoring.
The central limitation for interpreting efficacy is that the primary outcome’s numerical results are not reported in the registry record. Without the means and standard errors, the direction, magnitude, and precision of tremor measurements cannot be assessed. The record therefore does not establish whether scores changed, how they varied over time, or whether the two formulations differed.
Even if numerical within-participant changes were available, the design would remain important. A single-group, unblinded study without a randomized control comparison cannot separate a treatment effect from other possible influences on measured tremor. The record does not report a statistical contrast or inferential results that would address a formulation comparison.
General statistical context: an observed change is a description of data, while a causal conclusion requires a design and analysis that address alternative explanations. Precision measures help show uncertainty in an estimate, but they do not resolve design limitations. The registry entry supplies the endpoint definition and some design information, but not the results and analysis details needed for a full statistical assessment.
Accordingly, the appropriate reading is limited to what the record documents: the study’s planned measurements and reported safety entries. It does not support a conclusion that 1-octanol did or did not reduce tremor, or that one formulation was better than another.
13. Why This Trial Matters Statistically
This study is statistically notable for focusing on a relative, repeated-measures outcome rather than a single raw measurement. Normalizing tremor amplitude to baseline gives the endpoint a clear reference: 1 represents baseline severity, while lower values indicate tremor reduction. Measurements over the listed post-dose times can, in principle, describe how the outcome varies over the observation period.
The design also illustrates why the analysis population matters. The primary endpoint applies to participants who received both formulations in Part B, whereas the overall enrollment is listed separately. The registry record does not report the size of that endpoint population or the number with measurements at each time. Those details are needed to judge how much information supports the estimated means.
At the same time, the absence of a randomized control group limits the questions that the design can answer. Repeated measurements can characterize outcomes among participants studied, but they do not by themselves identify a causal effect. The unblinded status may also matter when measurement or study conduct could be influenced by knowledge of treatment, although the registry record does not describe whether or how that issue was addressed.
Finally, the record makes a distinction between observed outcomes and planning illustrations important. A sample-size calculation based on an observed effect can be useful for planning future work, but it is not observed power and does not replace reporting the study’s estimates and uncertainty. Here, the numerical primary outcome results and analysis details are not reported in the registry record. The design and endpoint can be described, but the clinical signal and its precision cannot be evaluated from the available results.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The registry record gives limited detail about the two-phase inpatient design. The brief description is incomplete, so the procedures within each phase, the timing of formulation administration, and the relationship between phases are not reported in the registry record. The record identifies a primary population that received both formulations in Part B but does not state how many participants met that definition.
Numerical primary-outcome results are not reported in the registry record. The means, standard errors, and time-specific participant counts are therefore unavailable for assessing the observed tremor pattern or its precision. The record also does not report the statistical model, the approach to repeated measurements, or the handling of missing data.
The design is unblinded and single-group, with allocation listed as not applicable. A randomized control comparison is not described. Consequently, the registry entry does not support a controlled estimate of treatment effect or a like-for-like comparison with an independent control group. The record does not report whether any adjustment was made for baseline or other characteristics.
The registry also does not report a detailed statistical analysis plan, multiplicity strategy, sample-size justification, or interim-analysis plan. The planning illustration is described as based on an observed effect and should not be mistaken for the original study design or observed power. The safety entries lack details about event timing and causal assessment, and deaths are not reported in the registry record. These omissions constrain interpretation of both the outcome and safety information.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Clinical Trial Characterizing the Bioavailability of 1-Octanol in Adults With Ethanol-responsive Essential Tremor — NCT00102596.
- Registry record last updated 2012-02-01; status last verified 2012-01.
- Results first posted 2012-02-01.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
The registry describes a completed Phase 2, unblinded, single-group study of 1-octanol in adults with ethanol-responsive essential tremor. Enrollment is listed as 21. The primary outcome is mean normalized right-hand tremor amplitude after both formulations of 64 mg/kg in Part B, measured by spirography at the specified post-dose times. A normalized score of 1 represents baseline severity, and lower scores indicate tremor reduction.
The record also lists secondary outcomes related to plasma octanoic acid, heart rate, and PR and QTc intervals, along with serious and other adverse-event counts. Numerical primary-outcome results, detailed analysis methods, missing-data procedures, and a multiplicity strategy are not reported in the registry record. The design and endpoint can be described, but the magnitude and precision of the tremor results cannot be assessed from the posted results.